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Objective: A comparative assessment of treatment alternatives for T1N0 anal canal cancer has never been conducted. We compared the outcomes associated with the treatment alternatives—chemoradiotherapy (CRT), radiotherapy (RT), and surgery or ablation techniques (surgery/ablation)—for T1N0 anal canal cancer.

Materials and Methods: This retrospective cohort study was conducted using the Surveillance, Epidemiology and End Results (SEER) registries linked with Medicare longitudinal data (SEER-Medicare database). Analysis included 190 patients who were treated for T1N0 anal canal cancer using surgery/ablation (n=44), RT (n=50), or CRT (n=96). The outcomes were reported in terms of survival and hazards ratios using Kaplan-Meier and Cox proportional hazards modeling, respectively; lifetime costs; and cost-effectiveness measured in terms of incremental cost-effectiveness ratio, that is, the ratio of the difference in costs between the 2 alternatives to the difference in effectiveness between the same 2 alternatives.

Results: There was no significant difference in the survival duration between the treatment groups as predicted by the Kaplan-Meier curves. After adjusting for patient characteristics and propensity score, the hazard ratio of death for the patients who received CRT compared with surgery/ablation was 1.742 (95% confidence interval, 0.793-3.829) and RT was 2.170 (95% confidence interval, 0.923-5.101); however, the relationship did not reach statistical significance. Surgery/ablation resulted in lower lifetime cost than RT or CRT. The incremental cost-effectiveness ratio associated with CRT compared with surgery/ablation was $142,883 per life year gained.

Conclusions: There was no statistically significant difference in survival among the treatment alternatives for T1N0 anal canal cancer. Given that surgery/ablation costs less than RT or CRT and might be cost-effective compared with RT and CRT, it is crucial to explore this finding further in this era of limited health care resources.

*Department of Health Services Research, Management and Policy, University of Florida, Gainesville, FL

Departments of †Health Services Research

‡Radiation Oncology

∥Surgical Oncology, The University of Texas MD Anderson Cancer Center

§Department of Medicine, Section of Infectious Disease, Baylor College of Medicine

#The University of Texas Health Science Center of Houston School of Public Health, Houston, TX

¶Department of Health Services Administration, University of Maryland School of Public Health, College Park, MD

Supported by US National Cancer Institute (R01 CA163103), The Janice Davis Gordon Postdoctoral Fellowship in Colorectal Cancer Prevention, and the National Institutes of Health through The University of Texas MD Anderson Cancer Center Support Grant (CA016672), and the Duncan Family Institute.